FDA Approves Adult-Onset Still's Disease Treatment

The US Food and Drug Administration (FDA) has approved canakinumab (Ilaris) injections for the treatment of Active Still’s disease, including Adult-Onset Still’s Disease (AOSD).

The FDA previously approved the treatment for Systemic Juvenile Idiopathic Arthritis (SJIA) in patients at least 2 years.

Adult-Onset Still’s Disease is a rare and serious autoinflammatory disease without a known origin. Autoinflammatory disease are caused by abnormalities of the immune system that trigger an inflammatory response that can damage the body’s own tissues.

In AOSD, there is significant overlap with SJIA, including fever arthritis, rash and elevated markers for inflammation.

There is a well-established role in the 2 diseases of interleukin-1 (IL-1), a type of cytokine important in regulating the immune system.

Canakinumab blocks the effects of IL-1 and suppresses inflammation in patients with the autoinflammatory disorders.

The FDA approval is based on well-established safety and efficacy of the drug in AOSD using comparable pharmacokinetic exposure and extrapolation of established efficacy of canakinumab in patients with SJIA, as well as the safety of canakinumab in patients with AOSD and other diseases.

Some common side effects include colds and upper respiratory tract infections, abdominal pain, and injection-site reactions. The prescribing information includes a warning for potential increased risk of serious infections because of IL-1 blockades.

Treatment with immunosuppressants may increase the risk of malignancies. Patients are advised not to receive live vaccinations during treatment. Macrophage activation syndrome is also a life-threatening risk for patients with rheumatic conditions.

"Prior to today's approval, patients had no FDA-approved treatments for their disease, which can include symptoms such as painful arthritis, fevers and rash," Nikolay Nikolov, MD, acting director of the Division of Rheumatology and Transplant Medicine in the FDA's Center for Drug Evaluation and Research, said in a statement. "Today's approval provides patients with a treatment option."